PB2511: CLINICAL COMPLICATIONS AMONG PATIENTS WITH SICKLE CELL DISEASE WITH RECURRENT VASO-OCCLUSIVE CRISES IN GERMANY
Authors: Chuka Udeze, Colin Kunzweiler, Nanxin LI, Jessica Baldwin, Petra Tuzin, Sebastian Dietmar Barth, Céline Vetter, Silvia Dombrowski, Elena Georgiadou-Schmidt, Roland Meisel
Journal: HemaSphere (2023)

Topic: 26. Sickle cell disease Background: Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by abnormal expression of sickle hemoglobin, which leads to vaso-occlusive crises (VOCs), significant morbidity, and early mortality due to end-organ damage. SCD complications are multifactorial and driven by vaso-occlusion, hemolysis, and vasculopathy associated with the disease. Patients with SCD require lifelong supportive care and intensive treatments. Current non-curative treatments include hydroxyurea, crizanlizumab, voxelotor, transfusions with or without iron chelation therapy, antibiotics, and pain-relieving medications to reduce the frequency/severity of pain crises, prevent infections, and relieve pain in patients with severe disease. Aims: To describe clinical complications in patients with SCD with recurrent VOCs in Germany. Methods: This longitudinal, retrospective cohort study utilized statutory health insurance records (Betriebskrankenkasse data source) in Germany to identify patients with a diagnosis for SCD between January 1, 2010, and December 31, 2018 (eligibility period). Eligible patients with SCD were required to have ≥2 VOCs/year in any 2 consecutive years during the eligibility period. A VOC was defined as SCD with pain crisis, priapism, or acute chest syndrome. Patients were required to have data for ≥1 year before and after their index date (the second VOC in the second year of the 2 consecutive years). Patients who had received a hematopoietic stem cell transplant were excluded. Patients were followed from index until the earliest of death, deregistration due to patient leaving the data source, or end of study period (December 31, 2019). Demographics were assessed at index. Acute and chronic complications (proportion of total population) were summarized descriptively during follow-up. Results: In total, 84 patients with SCD with recurrent VOCs were included. Their mean age at index was 40.1 (standard deviation [SD]=26.0) years, and 37 (44.0%) were female. Average length of follow-up was 6.0 years. Patients experienced an average of 4.0 (SD=3.9) VOCs per-patient-per-year during follow-up. The most common acute complications (>10% prevalence) were multisystem organ failure (40.5%), infections (23.8%), stroke (15.5%), and gallstones (13.1%). The most common chronic complications (>10% prevalence) were retinal disorders/retinopathy (45.2%); mental illness (depression or anxiety [31.0%]); cardiovascular complications (25.0%); chronic pain (22.6%); renal complications (19.1%); hyposplenism (15.5%); bone and joint problems (14.3%); hepatobiliary complications (13.1%); malignancies (11.9%); and infertility (10.7%). Summary/Conclusion: Despite the best available care, patients with SCD with recurrent VOCs experience substantial burden due to SCD-related clinical complications, highlighting the need for innovative therapies in this space. Keywords: Complications, Hemoglobinopathy, Sickle cell disease